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Experiment: Second site validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain

PI:   Jason D Heaney, PhD 
 

Validation of delivery of AAV custom designed to cross the blood-brain barrier for CRISPR/Cas9 editing. Editing detected and quantified in brain by generation of tdTomato fluorescent protein signal from Ai9 reporter mice
Delivery Assays: - Identification of of GFP-positive nuclei (green)
Editing Assay: - Qualitative assesment of tdTomato-negative cells throughout the entire brain
- Quantitative assessment of tdTomato-positive cells per DAPI-stained nucleus in a specific brain region
- Qualitative assesment of prevalence of tdTomato-positvie cells throughout the entire brain

Organ System Overview


 
Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Males
Arm1a: BI28 Neuron Specific Promoter, Experimental Animals, Females
Arm 1b: BI28 Neuron Specific Promoter, Positive Delivery Control Animals, Males
Arm 1b: BI28 Neuron Specific Promoter, Positive Delivery Control Animals, Females
Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Males
Arm 2a: BI28 Astrocyte Specific Promoter, Experimental Animals, Females
Arm 2b: BI28 Astrocyte Specific Promoter, Positive Delivery Control Animals, Males
Arm 2b: BI28 Astrocyte Specific Promoter, Positive Delivery Control Animals, Females
Arm 3: Negative Control Saline, Males
Legend:  
Delivery Efficiency
Editing Efficiency
Target Tissue
Not Available
 

 
Analyze Data Sets Available for this Experiment
Nervous
  Brain (neuron)  (TARGET)
  Cerebral cortex (astrocyte)  (TARGET)
  Dorsal plus ventral thalamus (astrocyte)  (TARGET)
  Brain (astrocyte)  (TARGET)
  Brain (unspecified)